A Study to Assess the Effectiveness and Safety of Pacritinib in Patients With VEXAS Syndrome (PAXIS)
Trial · NCT06782373
Registered outcomes15
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
1 to 15 of 15
Download this page as JSON| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Overall Response Rate (ORR), defined as achieving Partial Clinical Response or better at any time during the double-blind treatment period. | up to End of Week 24 | Difference in the proportion of participants achieving ORR for the pairwise comparison of pacritinib dose A vs. placebo and pacritinib dose B plus placebo vs. placebo (Phase 2a) and selected pacritinib dose compared to placebo (Phase 2b) |
| Secondary | Overall Clinical Response (OCR), defined as achieving Clinical Response or better at any time during the double-blind treatment period | up to End of Week 24 | Difference in the proportion of participants achieving OCR for the pairwise comparison of pacritinib dose A vs. placebo and pacritinib dose B plus placebo vs. placebo (Phase 2a) and selected pacritinib dose compared to placebo (Phase 2b) |
| Secondary | Number of flare-free days with GC dose ≤10 mg | up to End of Week 24 | |
| Secondary | Proportion of participants on each arm achieving each Best Response category (Clinical Biochemical Response, Clinical Response, Partial Clinical Response, Stable Disease, or Non-response). | up to End of Week 24 | |
| Secondary | Hematologic Improvement - Erythroid | up to End of Week 24 | Erythroid (HI-E) at any time among participants with baseline hemoglobin \<10 g/dL per modified International Working Group (IWG) criteria |
| Secondary | Hematologic Improvement - Platelets | up to End of Week 24 | Platelets (HI-P) at any time among participants with baseline platelet count \<100 × 10\^9/L per modified IWG criteria |
| Secondary | Change in health-related quality of life (QOL) as measured by Patient-Reported Outcomes Measurement Information Systems (PROMIS) fatigue short form | up to End of Week 24 | The PROMIS short form domains of fatigue 4a will be summarized, raw score from 4 to 20 (T-score from 33.7 to 75.8). A higher score is associated with more fatigue. |
| Secondary | Change in health-related QOL as measured by PROMIS physical function short form | up to End of Week 24 | The PROMIS short form domains of physical function 4a will be summarized, raw score from 4 to 20 (T-score from 22.5 to 57.0). A higher score is associated with better physical function. |
| Secondary | Change in health-related QOL as measured by PROMIS sleep disturbance short form | up to End of Week 24 | The PROMIS short form domains of sleep disturbance 4a will be summarized, raw score from 4 to 20 (T-score from 32.0 to 73.3). A higher score is associated with more sleep disturbance. |
| Secondary | Change in health-related QOL as measured by the 36-item short form (SF) Survey | up to End of Week 24 | The SF-36 Survey measures eight domains of health status; physical functioning, role limitations due to physical health, role limitations due to emotional problems, energy/fatigue, emotional well-being, social functioning, pain, and general health. The values for each domain range from 0 to 100, with the higher score indicating a better health status. |
| Secondary | Change in health-related QOL as measured by Patient's Global Impression of Change (PGIC) response | up to End of Week 24 | The number and percentage of participants achieving PGIC response (reporting symptoms as "much" or "very much" improved). |
| Secondary | Mean plasma concentration of pacritinib | up to Week 24 (pre-dose only) | Mean plasma concentrations (pre-dose and post-dose) will be determined for each pharmacokinetic sampling timepoint. |
| Secondary | Change in pharmacodynamic (PD) inflammatory biomarker: C-reactive protein (CRP) | up to 30-day Post-End of Trial | |
| Secondary | Change in PD inflammatory biomarker: erythrocyte sedimentation rate (ESR) | up to 30-day Post-End of Trial | |
| Secondary | Change in PD inflammatory biomarker: ferritin | up to 30-day Post-End of Trial |
Source ClinicalTrials.govtrials-2026-11-12-0.5.0
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this list from software.
Twenty rows a page, as JSON. No key.
GET https://conductscience.org/api/trials/v1/NCT06782373/registered